New Phase 3 Data Reinforce Long-Lasting Protection from Bleeding for Patients with Hemophilia A and B

New Phase 3 Data Reinforce Long-Lasting Protection from Bleeding for
Patients with Hemophilia A and B
– Recombinant Fc fusion proteins show potential to transform care by providing
long-lasting protection from bleeding with fewer injections than the current
standard of care –
Business Wire
WARSAW, Poland -- February 8, 2013
Biogen Idec (NASDAQ: BIIB) and Swedish Orphan Biovitrum (Sobi) (STO: SOBI)
released data that confirmed the ability of investigational recombinant
factors VIII Fc fusion protein (rFVIIIFc) and IX Fc fusion protein (rFIXFc) to
provide long-lasting protection from bleeding with fewer injections than are
required with the current standard of care for people with hemophilia. The
data, from the largest phase 3 registrational studies conducted in hemophilia
to date, were presented this week at the 6th Annual Congress of the European
Association for Haemophilia and Allied Disorders (EAHAD).
The studies compared the pharmacokinetic activity of rFVIIIFc for hemophilia A
and rFIXFc for hemophilia B to currently available treatments. In the studies,
the long-lasting candidates stayed active in the body longer, enabling study
participants to prevent bleeding with less frequent injections than are
required with the current standard of care. In the A-LONG study, patients with
hemophilia A were able to use once to twice weekly prophylactic (preventative
dosing) injections of rFVIIIFc while maintaining low bleeding rates. In the
B-LONG study, rFIXFc allowed patients with hemophilia B to use prophylactic
injections every one to two weeks with low bleeding rates.
“Data from these phase 3 trials demonstrate a potential to transform the
treatment of hemophilia by offering long-lasting protection from bleeding
while meaningfully reducing treatment burden associated with this rare
disease,” said Glenn Pierce, M.D., Ph.D., senior vice president of Global
Medical Affairs and chief medical officer of Biogen Idec’s hemophilia
therapeutic area. “Less frequent injections may help more people with
hemophilia adhere to a preventative treatment schedule, which can help prevent
the long-term health consequences associated with treating a bleed after it
occurs.”
The current standard of care for hemophilia A and B requires frequent
injections, which are a burden for patients. Prophylactic treatment for
hemophilia A typically requires injections three times per week or every other
day, and injections 2-3 times per week for the treatment of hemophilia B,
according to the National Hemophilia Foundation's Medical and Scientific
Advisory Council guidelines. People with severe hemophilia who do not follow a
prophylactic injection schedule remain vulnerable to bleeding that can cause
irreversible joint damage and life-threatening hemorrhages.
Recombinant FVIIIFc and recombinant FIXFc were developed using Fc fusion
technology, which has safely been used in FDA-approved medicines for more
than a decade. Biogen Idec and Swedish Orphan Biovitrum applied Fc fusion
technology in hemophilia for the first time with the goal of making clotting
factors last longer and reduce the burden of injections for patients and their
families.
About the A-LONG (rFVIIIFc) Data at EAHAD
The A-LONG results confirm the long-lasting characteristics of rFVIIIFc;
specifically, the data show that rFVIIIFc stays in the body for 50 percent
longer than Advate^® [antihemophilic factor (recombinant), plasma/albumin-free
method], the most frequently used factor VIII therapy. In the trial, the
terminal half-life for rFVIIIFc was 19 hours compared to 12 hours for Advate.
Other measures of rFVIIIFc’s activity in the body reinforce its long-lasting
characteristics: the mean time for maintaining a clotting factor activity
level associated with less bleeding (time to 1 percent) was approximately 5
days for rFVIIIFc compared to 3.5 days for Advate and the average rate at
which rFVIIIFc was cleared from the body was 2.0 mL/hr/kg compared with 3.0
mL/hr/kg for Advate. In the study’s individualized prophylaxis arm, patients
received rFVIIIFc at a median dosing interval of 3.5 days and a median weekly
dose of 78 IU per kg to prevent bleeding, which compares favorably to the
recommended dose for the standard of care. Nearly one-third of patients were
able to achieve every 5 day dosing in this arm. Overall, the A-LONG data
indicate that rFVIIIFc has the potential to become the first product to offer
hemophilia A patients long-lasting protection from bleeding with less frequent
dosing than the current standard of care.
The A-LONG data were presented in the late-breaking oral abstract session and
in poster 104, “Phase 3 clinical study of recombinant FC fusion factor FVIII
(rFVIIIFc) demonstrated safety, efficacy, and improved pharmacokinetics
(A-LONG).”
About the B-LONG (rFIXFc) Data at EAHAD
The B-LONG results confirm the long-lasting characteristics of rFIXFc;
specifically, the data show that rFIXFc stays in the body for more than twice
as long as BeneFIX^® [Coagulation Factor IX (Recombinant)], the only
recombinant factor IX therapy currently approved for prophylactic use. The
terminal half-life for rFIXFc was 82 hours compared to 34 hours for BeneFIX.
Other measures of rFIXFc’s activity in the body reinforce its long-lasting
characteristics: the mean time for maintaining a normal clotting factor
activity level (time to 1 percent) was 11 days for rFIXFc compared to 5 days
for BeneFIX and the average rate at which rFIXFc was cleared from the body was
3.2 mL/hr/kg compared with 6.3 mL/hr/kg for BeneFIX. All patients in the
individualized interval prophylaxis arm of the study were able to go at least
one week between rFIXFc injections and 50 percent were able to go 14 days or
longer before needing another dose to prevent bleeding. The median weekly dose
was 45 IU per kg, comparable to the recommended dose for the current standard
of care. Overall, the B-LONG data support the potential for rFIXFc to become
the first product to offer hemophilia B patients long-lasting protection from
bleeding with a more convenient injection schedule than the current standard
of care.
The B-LONG data were presented in poster 115, “Safety, efficacy, and improved
pharmacokinetics (PK) demonstrated in a phase 3 clinical trial of extended
half-life recombinant FC fusion factor IX (B-LONG).”
Importantly, the difference in the duration of activity of rFVIIIFc and rFIXFc
was expected and consistent with the differences between the natural clotting
factors that these products augment. Fc Fusion technology extends FVIII and
FIX differently based on the biological differences in hemophilia A and B. The
length of time that FVIII stays active is dictated by its own duration as well
as that of the blood protein that it binds to, known as von Willebrand factor.
The activity of FIX is not restricted in this way.
“These new data support the application of Fc fusion technology in hemophilia,
using a naturally occurring pathway to delay the breakdown of factor in the
body and cycle it back into the bloodstream,” said Birgitte Volck, M.D.,
Ph.D., senior vice president & chief medical officer of Sobi. “While Fc fusion
has been used in medicines for more than a decade, rFVIIIFc and rFIXFc are the
first investigational therapies to use the technology to successfully extend
the half-lives of clotting factors, which could offer protection from bleeding
while reducing the burden of injections for people with hemophilia.”
About the A-LONG Study and the rFVIIIFc Program
A-LONG was a global, open-label, multi-center phase 3 study that evaluated the
efficacy, safety and pharmacokinetics of intravenously-injected rFVIIIFc in
165 male patients aged 12 years and older. The study results, first announced
in October 2012, showed that rFVIIIFc was effective in the control and
prevention of bleeding, routine prophylaxis and perioperative management, with
low single-digit median annualized bleeding rates using individualized and
weekly prophylactic regimens. Overall, 98 percent of bleeding episodes were
controlled by one or two injections of rFVIIIFc. Recombinant FVIIIFc was
generally well-tolerated and no inhibitors to rFVIIIFc were detected. The most
common adverse events (incidence of ≥5 percent) occurring outside of the
perioperative management period were nasopharyngitis, arthralgia, headache and
upper respiratory tract infection. No serious adverse events were assessed to
be related to the therapy by the investigators.
Ongoing clinical studies of rFVIIIFc include Kids A-LONG, for
previously-treated children with hemophilia A under age 12, and ASPIRE, for
patients who completed the A-LONG study or who complete the Kids A-LONG study.
About the B-LONG Study and the rFIXFc Program
B-LONG was a global, open-label, multi-center phase 3 study that evaluated the
efficacy, safety and pharmacokinetics of intravenously-injected rFIXFc in 123
male patients aged 12 years and older. The study results, first announced in
September 2012, showed that rFIXFc was effective in the control and prevention
of bleeding, routine prophylaxis, and perioperative management, with low
single-digit median annualized bleeding rates using individualized
prophylactic regimens at a median dosing interval of 14 days. More than 90
percent of bleeding episodes were controlled by a single injection of rFIXFc.
Recombinant FIXFc was generally well-tolerated and no inhibitors to rFIXFc
were detected. The most common adverse events (incidence of ≥5 percent)
occurring outside of the perioperative management arm (i.e., Arms 1, 2 and 3,
but not Arm 4) were nasopharyngitis, influenza, arthralgia (joint pain), upper
respiratory infection, hypertension and headache. One serious adverse event,
obstructive uropathy in the setting of hematuria, was assessed to be possibly
related to therapy by the investigator. The patient continued rFIXFc treatment
and the event resolved with medical management.
Ongoing clinical studies of rFIXFc include Kids B-LONG, for previously treated
children with hemophilia B under age 12, and B-YOND, for patients who
completed the B-LONG study or who complete the Kids B-LONG study.
About the Fc Fusion Technology Platform
Recombinant FVIIIFc and recombinant FIXFc are clotting factors developed using
Biogen Idec’s novel and proprietary monomeric Fc fusion technology, which
makes use of a naturally occurring pathway that delays the breakdown of factor
in the body and cycles it back into the bloodstream, enabling it to remain in
the body longer following an injection. Fc fusion technology is used in seven
FDA-approved products for the long-term treatment of chronic diseases
including rheumatoid arthritis, psoriasis and platelet disorders.
About Hemophilia
Hemophilia is a rare, inherited disorder in which the ability of a person’s
blood to clot is impaired. Hemophilia A is caused by reduced or no Factor VIII
protein, whereas hemophilia B is caused by reduced or no Factor IX protein.
Both proteins are needed for normal blood clotting. Hemophilia A and
hemophilia B occur in about one in 5,000 and one in 25,000 male births,
respectively. People with hemophilia need injections of clotting factors to
restore the coagulation process and prevent frequent bleeds that could
otherwise lead to pain, irreversible joint damage and life-threatening
hemorrhages. The Medical and Scientific Advisory Council of the National
Hemophilia Foundation recommends prophylaxis as the optimal therapy for people
with severe hemophilia A and severe hemophilia B. Currently, prophylaxis for
hemophilia A typically requires injections three times per week or every other
day to maintain a sufficient circulating level of clotting factor, while
prophylaxis in hemophilia B typically requires injections two to three times
per week.
About the Biogen Idec and Sobi Collaboration
Biogen Idec and Sobi are partners in the development and commercialization of
rFVIIIFc and rFIXFc. Biogen Idec leads development, has manufacturing rights,
and has commercialization rights in North America and all other regions
excluding the Sobi territory. Sobi has the right to opt in to assume final
development and commercialization in Europe, Russia, the Middle East and
Northern Africa.
About Biogen Idec
Through cutting-edge science and medicine, Biogen Idec discovers, develops and
delivers to patients worldwide innovative therapies for the treatment of
neurodegenerative diseases, hemophilia and autoimmune disorders. Founded in
1978, Biogen Idec is the world's oldest independent biotechnology company.
Patients worldwide benefit from its leading multiple sclerosis therapies, and
the company generates more than $5 billion in annual revenues. For product
labeling, press releases and additional information about the company, please
visit www.biogenidec.com.
About Sobi
Sobi is an international healthcare company dedicated to bringing innovative
therapies and services to improve the lives of rare disease patients. The
product portfolio is primarily focused on inflammation and genetic diseases,
with three late stage biological development projects within hemophilia and
neonatology. Sobi also markets more than 40 products for companies in the
specialty and rare disease space. In 2011, Sobi had revenues of SEK 1.9
billion and around 500 employees. The share (STO: SOBI) is listed on NASDAQ
OMX Stockholm. More information is available at www.sobi.com.
Safe Harbor
This press release contains forward-looking statements, including statements
about the commercialization and impact of long-lasting hemophilia therapies.
These statements may be identified by words such as "believe," "expect,"
"may," "plan," "potential," "will" and similar expressions, and are based on
our current beliefs and expectations. Drug development and commercialization
involve a high degree of risk. Factors which could cause actual results to
differ materially from our current expectations include the risk that
unexpected concerns may arise from additional data or analysis, regulatory
authorities may require additional information or further studies, or may fail
to approve or may delay approval of our drug candidates, or we may encounter
other unexpected hurdles. For more detailed information on the risks and
uncertainties associated with Biogen Idec’s drug development and
commercialization activities, please review the Risk Factors section of Biogen
Idec’s most recent annual or quarterly report filed with the Securities and
Exchange Commission. Any forward-looking statements speak only as of the date
of this press release and we assume no obligation to update any
forward-looking statements, whether as a result of new information, future
events or otherwise.
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Contact:
BIOGEN IDEC CONTACT:
Media Contact:
Jeff Boyle, +1-781-464-3260
or
Investor Relations Contact:
Kia Khaleghpour, +1-781-464-2442
or
SWEDISH ORPHAN BIOVITRUM CONTACT:
Media Contact:
Birgitte Volck, +45 4 012 2310/+46 8 697 2188
or
Analyst/Investor Contact:
Jorgen Winroth, +46 8 697 2000/+1-212-579-0506